Glioblastoma: clinical success
Glioblastoma is detected annually in approximately 15,000 patients in the United States and always leads to a fatal outcome. The difficulty in developing effective drugs against this tumor lies in the presence of a blood-brain barrier that prevents them from reaching the brain.
An early clinical study on patients with glioblastoma showed that the experimental preparation of spherical nucleic acid NU-0129, developed by scientists at Northwestern University, is able to penetrate the blood-brain barrier and cause the death of tumor cells.
The phase 0 study was conducted with eight people with recurrent glioblastoma at the Robert H. Lurie Cancer Center at Northwestern University. In phase 0, researchers use a small dose of the drug to make sure it is safe for humans before they start using it in higher doses in larger clinical trials.
All participants planned surgical removal of the tumor; before the operation, they were injected intravenously with NU-0129. After the tumor was removed, the team examined the samples and determined that the drug overcomes the blood-brain barrier and triggers apoptosis in cancer cells. Previously, a similar effect was noted in animal studies and has now been confirmed in humans.
NU-0129 is a preparation of spherical nucleic acid, which consists of nuclei of gold nanoparticles covalently bound to radially oriented and tightly packed small interfering RNAs (siRNAs). This is the first spherical nucleic acid preparation developed for systemic use. The three-dimensional design allows you to penetrate tumor cells, correct genes and make them vulnerable.
The new spherical nucleic acid platform can be used to develop drugs for the treatment of other neurological diseases, including Alzheimer's, Huntington's and Parkinson's diseases, similarly disabling the genes that cause these diseases.
Article by P.Kumthekar et al. A first-in-human phase 0 clinical study of RNA interference–based spherical nucleic acids in patients with recurrent glioblastoma is published in the journal Science Translational Medicine.
Aminat Adzhieva, portal "Eternal Youth" http://vechnayamolodost.ru Based on materials from Northwestern University: New Spherical Nucleic Acid 'Drug' Kills Tumor Cells in Humans With Glioblastoma.